2018 Achillin Pharmaceuticals. All rights reserved. NASDAQ:ACHN
Cautinary Nte Regarding Frward-Lking Statements This presentatin includes frward-lking statements within the meaning f the Private Securities Litigatin Refrm Act f 1995 that are subject t risks, uncertainties and ther imprtant factrs that culd cause actual results t differ materially frm thse indicated by such frward-lking statements. Achillin may use wrds such as expect, anticipate, prject target, intend, plan, aim, believe, seek, estimate, can, culd, fcus, will, lk frward, cntinue, gal, strategy, may and similar expressins t identify such frward-lking statements. These frward-lking statements include statements abut Achillin and its business and prspects, including, withut limitatin, statements regarding drug discvery, research, clinical develpment, timing f anticipated clinical trials and clinical data fr ur prduct candidates, ur expectatins regarding the ptential safety, efficacy and clinical utility f ur prduct candidates, regulatry apprval prcesses, market pprtunities, strategic gals, ur previus cllabratin with Janssen in HCV, intellectual prperty, cmpetitin, and financial results. T the extent that statements cntained in this presentatin are nt descriptins f histrical facts, they are frward-lking statements reflecting management s current beliefs and expectatins. Varius imprtant factrs may cause differences between ur frward-lking statements and actual results, including withut limitatin, unexpected r unfavrable safety r efficacy data, lwer than expected enrllment rates in clinical trials, changes in the cmpetitive landscape fr ur prduct candidates, changes in the regulatry envirnment, changes in market cnditins r future demand fr ur prduct candidates, the inability t prtect ur intellectual prperty, ur freedm t perate under third party intellectual prperty, ur need fr future capital, the risk f litigatin r ther disputes, and general market and ecnmic cnditins. These and ther risks and uncertainties are described in the reprts filed by Achillin with the SEC, including its annual reprt n Frm 10-K and quarterly reprts n Frm 10-Q, and subsequent filings with the SEC frm time t time. Yu shuld read these reprts, including the Risk Factrs cntained in these reprts with the understanding that ur actual future results may be materially different frm what we currently expect. All frward-lking statements cntained in this presentatin speak nly as f the date heref, and Achillin undertakes n bligatin t update any f these statements, except as required by law. 2
Achillin s Value Prpsitin Established prf-f-mechanism with ACH-4471 fr the treatment f C3G Significant imprvement in prteinuria demnstrating preliminary prf-f-cncept (PC) Significant imprvement in cmplement bimarkers demnstrating target engagement Ptential disease-mdifying therapy in area where n current treatment available Established PC with ACH-4471 fr the treatment f PNH Gd tlerability bserved at 200mg TID dse after mre than six mnths f treatment Demnstrated bth clinical (hemglbin) and bimarker (C3 fragment depsitin n PNH erythrcytes) imprvements fr balanced ability t address bth EVH and IVH in patients Factr D platfrm represents an ral, ptentially disease-mdifying therapy in multiple diseases $353.5 millin at 9/30/17 in cash, cash equivalents, and interest receivable t supprt achievement f value-accreting milestnes 3
2018 Gals and Milestnes Near-term ACH-4471 Clinical Develpment Plan Cmpund Indicatin Anticipated Next steps ACH-4471 Next-Gen Cmpunds C3G & IC-MPGN 14-day Ph 2 Grup 2 results 3Q18 12-mnth pen label Ph 2: FPFD 1H18 and interim results 4Q18 6-mnth randmized duble-blind C3G Ph 2: FPFD 1H18 with full enrllment anticipated YE18 PNH Mntherapy Ph 2: Interim results 3Q18 Add-n trial Ph 2: FPFD 1H18 and interim results by YE18 XR Prgram Biavailability study interim results 1H18 ACH-5228 Interim Ph 1 results 2H18 Additinal Initiate Ph 1 by YE18 $353.5 millin in cash, cash equivalents, and interest receivable at 9/30/17 t supprt clinical expansin and factr D platfrm 4
Achillin s Pipeline PROGRAM DELIVERY DISCOVERY : PRECLINICAL CLINICAL COMPLEMENT FACTOR D PLATFORM Discvery DMPK & Safety IND-Enabling Phase 1 Phase 2 Phase 3 C3 Glmerulpathy (C3G) ACH-4471: Factr D Inhibitr Oral Parxysmal Ncturnal Hemglbinuria (PNH) ACH-4471: Factr D Inhibitr Oral Immune Cmplex-mediated Membranrliferative Glmerulnephritis (IC MPGN) ACH-4471: Factr D Inhibitr Oral AP-mediated diseases ACH-5228: Next-Generatin Factr D inhibitr Oral AP-mediated phthamlgy diseases Next-Generatin Factr D Inhibitr Ophthalmic fd: Factr D DMPK: Drug Metablism/Pharmackinetics AP: Alternative Pathway 5
Mechanism Matters: Factr D Inhibitin Lectin Pathway Alternative Pathway C1q C1r-C1s C4 MBL MASP-1,2 C3 fh Ba fh C3 Classical Pathway C4a C2b C2 C3 fd fb C3b C3b C3(H 2 O) Bb fd C3(H 2 O) B C3(H 2 O) fb C4b2a C3bBb Ba C3bB C3dg C3d C3c ic3b C3a fb C3b C5 C4b2a3b C5a C3bBb3b C6-9 FACTOR D A critical cntrl pint specifically within the AP TRIGGER POINT INHIBITOR Prevents amplificatin and mdulates dwnstream cmplement cascade Terminal Pathway C5b 6
FACTOR D INHIBITOR PORTFOLIO Unlcking the Brader Ptential f ACH-4471 PLANS FOR EXPANDING CLINICAL PROGRAM C3G & IC-MPGN PNH Phase 2: 14-day dsing Phase 2: 12 mnth dsing (pen-label) Phase 2: 6-mnth dsing in C3G (duble-blind, placeb cntrlled) Natural histry study: Onging study cnducted by Imperial Cllege f Lndn anticipated t enrll up t 400 patients glbally Phase 2: Expanding patient enrllment in nging mntherapy trial in untreated patients; currently 3 subjects remain n treatment with 200 mg TID Phase 2: Add-n trial t supprt switch-strategy fr patients with subptimal respnse t eculizumab We aim t pineer best-in-disease factr D inhibitin acrss multiple indicatins 7
FACTOR D INHIBITOR PORTFOLIO ACH-4471 Summary f Regulatry Status Regulatry develpment planning is fcused n US, UK, EU and Japan, in additin t clinical trials cnducted in New Zealand and Australia Open U.S. FDA INDs fr C3G and PNH Orphan drug designatin in U.S. and CHMP Scientific advice has been btained frm regulatry agencies including: Design f Phase 2 studies fr C3G and PNH Safety mnitring plan Guidance n clinical pharmaclgy and nnclinical prgrams needed t supprt registratin Preliminary discussins and feedback n pivtal study endpints Cnfidential 8
ACH-4471 C3G / IC-MPGN: Phase 2 14-day Trial Interim Results and Next Steps
C3 GLOMERULOPATHY (C3G) A Rare Disease with N FDA-Apprved Treatment Renal Survival (%) C3G includes bth Dense Depsit Disease (DDD) and C3 glmerulnephritis (C3GN) Estimated prevalence f 8 12 peple affected per millin in majr markets 100 DDD AND C3GN IMPACT ON RENAL SURVIVAL Incidence rate f 1 2 per millin patients diagnsed with C3G n an annual basis 75 There are n apprved treatments indicated fr patients with C3G 50 Nn-specific treatment appraches include bld pressure cntrl and brad immunsuppressin 25 Significant unmet medical need as nearly half f C3G patients prgress t end-stage renal disease 30-50% prgress t ESRD within 10 years 0 0 10 20 30 40 Years frm Diagnsis C3GN (n=49) DDD (n=26) Greater than 50% f patients experience disease recurrence pst renal transplant, with a 50% chance f graft lss Barbur et al. (2015); NICE C3G Evidence Summary (2015); Surces: Servais et al (2013); Medjeral-Thmas et al (2014); Data n File. Achillin Pharmaceuticals. 2016. 10
IMMUNE COMPLEX-MEDIATED MEMBRANOPROLIFERATIVE GLOMERULONEPHRITIS (IC-MPGN) A Rare Disease with N FDA-Apprved Treatment Primary IC-MPGN is believed t be a renal disease assciated with AP hyperactivity Incidence and Prevalence Incidence rate similar t that f C3G: 1 t 2 per millin in develped areas (e.g. U.S., Eurpe) Accunts fr 4 10% f primary nephrtic syndrme in children Age Distributin: May ccur at any age Primary IC-MPGN ccurs mst cmmnly in lder children and yung adults (7 30 years) Clinical Presentatin Nephrtic syndrme, grss hematuria, and hypertensin Variable rate f prgressin tward renal failure Estimated 50% f patients prgress t end-stage renal disease within 10 years Nephrl Dial Transplant (2012) 27: 4288 4294; Silva s Diagntic Renal Pathlgy; Xin Jin (Jseph) Zhu et al - 2017; Pediatr Nephrl. 2010 Aug; 25(8): 1409 1418; Kidney Internatinal Supplements (2012) 2, 198 199; di:10.1038/kisup.2012.21; N Engl J Med 2012; 366:1119-1131; Silva s Diagntic Renal Pathlgy; Xin Jin (Jseph) Zhu, et al - 2017 11
C3 GLOMERULOPATHY (C3G) Phase 2 14-day Trial in Patients with C3G r IC-MPGN Clinical Trial Design Grup 1: 2 patients received ACH-4471 100mg TID x 14 days fllwed by 7-day taper Grup 2: Up t 8 additinal patients t receive ACH-4471 200mg TID x 14 days fllwed by 7-day taper Criteria Must have clinical and pathlgic diagnsis f C3G r IC-MPGN C3 must be <50% LLN with C4 >90% LLN Estimated glmerular filtratin rate cannt be < 45 ml/min/1.73m 2 Outcme Measures Changes in bimarkers f alternative pathway activity (AP) including: - C3 fragments and intact C3 levels, Bb, and Ba Prteinuria Pharmackinetic prfiles Clinical Trial Status Grup 1: Cmplete Grup 2: Plans t enrll 8 additinal patients nging 12
Nrmal Alternative Pathway and Kidney Histlgy C3 fh Ba fh C3 C3b C3(H 2 O) Bb C3(H 2 O) B C3(H 2 O) fb fd C3dg C3d C3c ic3b NORMAL GLOMERULAR FUNCTION N Prteinuria Nrmal GFR 13 NORMAL KIDNEY HISTOLOGY
Overactive Alternative Pathway and C3G Kidney Histlgy fh fh C3 C3 NeF Ba C3 C3b C3(H 2 O) Bb C3(H 2 O) B C3(H 2 O) fb fd Increased level C3d ic3b Decreased level C3dg C3c ABNORMAL GLOMERULAR FUNCTION Prteinuria Reduced GFR C3G DISEASE HISTOLOGY 14
Phase 2 14-day Trial in Patients with C3G r IC-MPGN Patient A: Baseline Characteristics Adult male with C3G; diagnsed in March 2017 Key cncmitant medicatins Prednislne Mycphenlate Enalapril Spirnlactne Disease characteristics at baseline prir t first dse: Prteinuria: Albumin t Creatinine rati (ACR) 259.3 mg/mml (ref range: 0 2.5) Urinalysis: 3+ prtein, 1+ bld, 27 RBCs/HPF, 19 WBCs/HPF BP: 126/72 egfr: 91 ml/min/1.73m 2 Fragment:Intact C3 rati: 0.1692 (ref range: 0.0085 0.0949) 15
Phase 2 14-day Trial in Patients with C3G r IC-MPGN Patient A: Significant Reductin in Prteinuria Observed ACR (mg/mml) 300 ALBUMIN TO CREATININE RATIO OVER TIME Dsing Taper Fllw up - Time-dependent decrease bserved in prteinuria as measured by ACR 250 200 150 - Greater than 50% reductin achieved during 14 days f treatment 100 50 PRE- DOSE 1 4 7 10 14 15 16 17 21 24 28 DAY - ACH-4471 demnstrated ptential early signs f clinical benefit 16
Phase 2 14-day Trial in Patients with C3G r IC-MPGN Patient A: Bimarker Imprvements Rati f Fragment/Intact C3 RATIO OF FRAGMENT/INTACT C3 0.2 0.18 0.16 0.14 0.12 0.1 0.08 0.06 0.04 0.02 0 Dsing Taper Fllw up 1 4 7 10 14 15 16 17 21 24 28 PRE- DOSE DAY - ACH-4471 prvided significant imprvement fragment:intact C3 rati - Decrease in C3 fragments is ptentially beneficial in C3G - Mechanistic apprach facilitates ability t address rt cause f AP-mediated diseases 17
Phase 2 14-day Trial in Patients with C3G r IC-MPGN Patient A: Bimarker Imprvements Ex viv Ba Frmatin (%) Relative t NHS 140 120 EX VIVO Ba FORMATION RELATIVE TO NORMAL HUMAN SERUM Dsing Taper Fllw up - ACH-4471 significantly decreased Ba levels, resulting frm cleavage f factr B by factr D 100 80 60 40 - Lwer levels f Ba suggest lwer levels f C3 cnvertase, resulting in lwer levels f C3 fragments 20 0 1 2 4 5 7 8 10 12 14 15 16 17 21 24 28 PRE- DOSE DAY 18
Phase 2 14-day Trial in Patients with C3G r IC-MPGN Patient A: Additinal Bimarker Imprvements Crrectin in cmplement bimarker prtein Bb 30% reductin in Bb level as cmpared t baseline T explre lcal changes in cmplement prteins in the kidney after dsing with ACH-4471 Rati f urinary Ba:creatinine mnitred Imprvements in multiple cmplement bimarkers demnstrated inhibitin f cmplement factr D by ACH-4471 Observed an 4.4-fld imprvement in Ba:creatinine rati as cmpared t baseline Ba prductin is a surrgate fr C3 cnvertase frmatin 19
Phase 2 14-day Trial in Patients with C3G r IC-MPGN Patient B: Baseline Characteristics Adult male with nephrtic syndrme; diagnsed with C3G in Nvember 2016 Key Cncmitant medicatins: Irbesartan Spirnlactne Disease characteristics n Day 1 prir t first dse: Prteinuria: Albumin t Creatinine rati (ACR) 580.3 mg/mml (ref range: 0 2.5) Urinalysis: 3+ prtein, 1+ bld, 42 RBCs/HPF, and 10 WBCs/HPF BP: 123/80 egfr: 73 ml/min/1.73m 2 Fragment:Intact C3 Rati: 0.1775 (ref range: 0.0085 0.0949) 20
Phase 2 14-day Trial in Patients with C3G r IC-MPGN Patient B: Significant Reductin in Prteinuria Observed ACR (mg/mml) 600 550 500 450 400 350 300 250 200 150 100 PRE- DOSE ALBUMIN TO CREATININE RATIO OVER TIME Dsing Taper Fllw up 1 4 7 10 14 15 16 17 21 24 28 35 DAY Time-dependent decrease bserved in prteinuria as measured by ACR - Greater than 50% reductin achieved during 14 days f treatment - ACH-4471 demnstrated ptential early signs f clinical benefit 21
Phase 2 14-day Trial in Patients with C3G r IC-MPGN Patient B: Bimarker Imprvements Rati f Fragment/Intact C3 0.18 0.17 0.16 0.15 0.14 0.13 0.12 0.11 0.1 0.09 0.08 PRE- DOSE RATIO OF FRAGMENT/INTACT C3 Dsing Taper Fllw up 1 4 7 10 14 15 16 17 21 24 28 DAY - ACH-4471 prvided significant - imprvement fragment:intact C3 rati - Decrease in C3 fragments is ptentially beneficial in C3G - Mechanistic apprach facilitates ability t address underlying cause f AP-mediated diseases 22
Phase 2 14-day Trial in Patients with C3G r IC-MPGN Patient B: Bimarker Imprvements Ex viv Ba Frmatin (%) Relative t NHS 160 140 120 100 80 60 40 20 Ex viv Ba FORMATION RELATIVE TO NORMAL HUMAN SERUM Dsing Taper Fllw up - ACH-4471 significantly decreased Ba levels, resulting frm cleavage f factr B by factr D - Lwer levels f Ba suggest lwer levels f C3 cnvertase, resulting in lwer levels f C3 fragments 0 PRE- DOSE 1 2 4 5 7 8 10 12 14 15 17 21 24 28 DAY 23
Phase 2 14-day Trial in Patients with C3G r IC-MPGN Patient B: Bimarker Imprvements Crrectin in cmplement bimarker prtein Bb Apprximately 50% reductin in cmplement prtein Bb as cmpared t baseline T explre lcal changes in cmplements in the kidney after dsing with ACH-4471 Rati f urinary Ba:creatinine mnitred Observed an 18.6-fld imprvement in Ba:creatinine rati ver baseline Imprvements in multiple cmplement bimarkers demnstrated inhibitin f cmplement factr D by ACH-4471 Ba prductin is a surrgate fr C3 cnvertase frmatin 24
Phase 2 14-day Trial in Patients with C3G r IC-MPGN Summary f Interim Prf-f-Cncept Data ACH-4471 was well-tlerated by bth patients N SAEs, discntinuatins due t AEs, r fevers PC established with 100 mg TID Preliminary POC established with 50% imprvement in prteinuria AP inhibitin cnfirmed based n changes in cmplement bimarkers Next steps Evaluating 200 mg TID ACH-4471 in Grup 2 Actively screening t enrll up t eight patients with C3G r IC-MPGN 25
C3 GLOMERULOPATHY (C3G) Patient-Fcused Drug Develpment (PFDD) Meeting Achillin was lead spnsr f externally-led PFDD meeting fcused n C3G in August 2017 First PFDD meeting fcused n a renal disease Led by the Natinal Kidney Fundatin and the FDA Gal is t understand the patient perspective PFDD meetings prvide an imprtant pprtunity t hear directly frm patients and caregivers Understand the impact f the disease n patients daily lives Input may infrm FDA s decisins thrughut the drug develpment prcess Patient experiences shared at the meeting highlight the unmet need and the urgency t develp transfrmative therapies 26
ACH-4471 Phase 2 PNH Three-mnth / Lng-term Extensin Trials Interim Results and Next Steps
PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) Factr D and Ptential Prtectin frm Intra- / Extra-vascular Hemlysis Type III PNH erythrcytes N treatment Anti-C5 therapy C3 fragment depsitin Intra-vascular hemlysis Breakthrugh and Extra-vascular hemlysis C3 fragment psnizatin via RES macrphages (liver, spleen) PNH RBCs treated with a fd inhibitr may be prtected frm bth intra- and extra-vascular hemlysis Factr D inhibitr Prtected Type III PNH erythrcytes Adapted frm Luzzatt L, Risitan AM, Ntar R. Haematlgica 2010;95(4):523 526. Harder M., et al. Bld. 2017; 129(8)970-80. 28
PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) A Rare Disease with Limited FDA-Apprved Treatment GOALS FOR INITIAL CLINICAL DEVELOPMENT Demnstrate prf-f-mechanism with a highly innvative apprach Limits C3 fragment depsitin n PNH red bld cells Reductin in plasma Bb levels Demnstrate prf-f-cncept by shwing clinical efficacy Reductin in LDH Increase in hemglbin Imprvement in fatigue scre (FACIT scre) Increase in PNH RBC clne size Elucidate PK/PD Early develpment prgram serves as a gateway t unlck brader ptential f ACH-4471 Understand plasma cncentratins f ACH-4471 necessary fr ptential efficacy Acceptable safety and tlerability prfile 29
Study Status and Interim Results Phase 2 Trial f ACH-4471 in Untreated PNH Patients Enrllment: 4 t 12 pts Three-mnth Dse Finding Lng-term Extensin KEY INCLUSION / EXCLUSION CRITERIA PATIENT D Classic PNH PATIENT C Classic PNH PATIENT B Aplastic Anemia / PNH PATIENT A Classic PNH PNH clne size > 10% Anemia (Hgb < 12 g/dl) Ttal days n therapy: 9 Days n 200mg TID: -- Ttal days n therapy: 44 Days n 200mg TID: 14 Ttal days n therapy: 126 Days n 200mg TID: 33 Ttal days n therapy: 132 Days n 200mg TID: 40 LDH > 1.5X ULN ANC > 1,000/ mm 3 Platelets > 50,000 μ/l Nrmal ALT Day 1 Day 28 Day 84 Alk Phs 1.5X ULN Part 1 Part 2 Lng-Term Objectives Reductin in LDH frm baseline Imprvements in Hgb, FACIT Increase PNH RBC clne size Investigatr determines clinical respnse t guide entry int Part 2 Investigatr assessment f benefit determines entry int extensin trial Extensin Study 30 Initial dse 100 mg TID. Prtcl subsequently amended t allw: - Newly enrlled patients t start at 150 mg TID - Intra-patient dse escalatin thrughut bth studies Interim data reprted by Achillin August 8, 2017
LDH (U/mL) Phase 2 Trial f ACH-4471 in Untreated PNH Patients Patient A: Classic PNH Male patient; diagnsed with PNH in 2011 after presentatin with dermal thrmbsis and hemlytic anemia - Otherwise healthy with active lifestyle; n transfusin requirements at baseline HgB (g/dl) LDH (U/L) FACIT PNH clne size (%) C3 fragment depsitin Current Value 14.1 272 49 43 Negative Baseline 11.7 1848 32 11 Negative Baseline Days n therapy 100mg TID * 13 days 150mg TID * 62 days 175mg TID * 17 days 200mg TID ** 40 days (last labs @ day 20) Status: Patient remains n 200 mg TID 2000 1848 16 1500 1000 11.7 14.1 14 12 10 HgB (g/dl) 500 0 2x ULN 1.5x ULN Hgb: hemglbin LDH: lactse dehydrgenase TID: three times daily 272 * Median values fr LDH and HgB shwn. ** Individual data pints shwn thrugh day 20 fr 200 mg TID grup. 8 6 Interim data reprted by Achillin August 8, 2017 31
LDH (U/mL) Phase 2 Trial f ACH-4471 in Untreated PNH Patients Patient C: Classic PNH Male patient; diagnsed with PNH in 2003 - Otherwise healthy with active lifestyle; n transfusin requirements at baseline (BL) 2000 1500 1000 500 0 Baseline Days n therapy 32 150mg TID * 13 days HgB (g/dl) LDH (U/L) 175mg TID ** 14 days FACIT PNH clne size (%) 200mg TID ** 14 days C3 fragment depsitin Current Value 12.8 551 36 43 Negative 2x ULN Baseline 11.7 1272 23 24 Negative 1272 11.7 1.5x ULN Hgb: hemglbin LDH: lactse dehydrgenase TID: three times daily Patient began taper n day 41 fllwing withdrawn cnsent. Patient Withdrew 12.8 551 * Median values fr LDH and HgB shwn. ** Individual data pints shwn thrugh day 14 fr 200 mg TID grup. 16 14 12 10 8 6 HgB (g/dl) Status: Patient reprted nncmpliance after initiatin f 200 mg TID dse Patient vluntarily withdrew cnsent fr reasns unrelated t safety n day 41 Interim data reprted by Achillin August 8, 2017
LDH (U/mL) Phase 2 Trial f ACH-4471 in Untreated PNH Patients Patient D: Classic PNH Male patient; diagnsed in 2012 with PNH - N histry f transfusin-dependence 2000 1500 1000 500 0 HgB (g/dl) LDH (U/L) FACIT PNH clne size (%) C3 fragment depsitin Current Value 12.4 504 n/a n/a n/a 2x ULN 1.5x ULN Baseline 12.0 899 n/a 36 Negative Baseline 12.0 899 Days n therapy 150mg TID * 9 days 12.4 504 Hgb: hemglbin LDH: lactse dehydrgenase TID: three times daily 175mg TID -- 200mg TID -- * Individual data pints shwn thrugh day 6 fr 150 mg TID grup. 16 14 12 10 8 6 HgB (g/dl) Status: Patient recently enrlled Currently receiving 150 mg TID dse and will be evaluated fr intra-patient dse escalatin Interim data reprted by Achillin August 8, 2017 33
LDH (U/mL) Phase 2 Trial f ACH-4471 in Untreated PNH Patients Patient B: Aplastic Anemia PNH Male patient diagnsed with AA in 2008; subsequently diagnsed with PNH in 2016 - Treated with ATG, ral prednisne and cyclsprine; ending in 2012 - Baseline marrw functin: platelets range 30-60K, ANC 0.7-1.5 and requires Q3-4 weekly RBC transfusins t maintain Hgb 8 g/dl 2000 1500 1000 500 0 HgB (g/dl) LDH (U/L) FACIT PNH clne size (%) C3 fragment depsitin Current Value 9.0 461 31 36 Negative 2x ULN Baseline 7.5 941 22 20 Negative Baseline 1.5x ULN Days n therapy 941 7.5 34 100mg TID * 13 days 150mg TID * 58 days 175mg TID * 22 days AA: Aplastic Anemia Hgb: hemglbin LDH: lactse dehydrgenase ANC: abslute neutrphil cunt TID: three times daily 200mg TID ** 33 days (last labs @ day 18) 9.0 461 * Median values fr LDH and HgB shwn. ** Individual data pints shwn thrugh day 18 fr 200 mg TID grup. 16 14 12 10 8 6 HgB (g/dl) Status: Patient remains n 200 mg TID Patient cntinued t receive RBC transfusins during therapy Interim data reprted by Achillin August 8, 2017
Measures f Clinical Efficacy & Safety in PNH Lactse dehydrgenase (LDH) Hemglbin (Hgb) C3 fragment depsitin Fatigue (FACIT scale) PNH RBC Clne Size Safety GOAL Clinically meaningful reductin in LDH GOAL Stabilize / increase hemglbin GOAL Observe n C3 fragment depsitin n PNH RBCs GOAL Imprvement ver time in bjective measures f patient fatigue GOAL Increase percentage f PNH RBC clnes frm baseline GOAL Favrable tlerability prfile Clinical data generated t date highlight the ptential rle f factr D inhibitin in PNH 35
Extended-Release Activities fr ACH-4471 ONGOING EXTENDED-RELEASE (XR) FORMULATION Objective Develp an extended release tablet frmulatin t allw fr: Optimized trugh expsures Reduced dsing frequency ACH-4471 has demnstrated high permeability, with animal and mdeling data reprting absrptin thrughut the GI tract Human biavailability study fr extended release tablet is planned by year-end 2017 36
FACTOR D INHIBITOR PORTFOLIO ACH-5228: Next-Generatin Oral Factr D Inhibitr Plasma Cnc. (ng/ml) Next-generatin factr D inhibitr platfrm can be leveraged t create additinal strategic ptins fr value creatin Structure alteratin in next-generatin factr D inhibitrs target imprvements in ptency and pharmackinetic prperties ACH-5228: Phase I single-ascending dse study initiated in December 2017 AP Hemlysis Cmpund ID IC 50 (nm) IC 90 (nm) ACH-4471 22 90 ACH-5228 9 34 ACH-5548 5 14 ACH-5628 17 75 ACH-5931 7 17 Plasma Cncentratins after an Oral Dsing in Beagle Dgs 10000 1000 100 10 1 ACH-4471 (20 mg/kg DN 5 mg/kg) ACH-5228 (5 mg/kg) ACH-5548 (5 mg/kg) ACH-5628(5 mg/kg) 0.1 0 6 12 18 24 Time (h) January 2017 37
2018 Achillin Pharmaceuticals. All rights reserved. NASDAQ:ACHN